The freedom and responsibility to make drug therapy decisions that are consistent with patient-care needs is a fundamental precept supported by ASHP. This activity is a professional duty of pharmacists not limited by language in Food and Drug Administration (FDA)-approved product labeling.
The prescribing, dispensing, and administration of FDA-approved drugs for uses, treatment regimens, or patient populations that are not reflected in FDA-approved product labeling often represent a therapeutic approach that has been extensively studied and reported in medical literature. Such uses are not indicative of inappropriate usage. Health-care professionals should appreciate the critical need for freedom in making drug therapy decisions and understand the implications of unlabeled uses. ASHP supports third-party reimbursement for FDA-approved drug products appropriately prescribed for unlabeled uses.
Definition of Unlabeled Use
The FDA approves drug products for marketing in the United States. Such a product approved for marketing is often termed an “FDA-approved drug.” FDA also approves each drug product’s labeling (container label, package insert, and certain advertising); the term “FDA-approved labeling” applies here. Drug uses that are not included in the indications or dosage regimens listed in the FDA-approved labeling are defined as “unlabeled uses.” For purposes of this document, unlabeled use includes the use of a drug product in (1) doses, (2) patient populations, (3) indications, or (4) routes of administration that are not reflected in FDA-approved product labeling.
It is important to recognize that FDA cannot approve or disapprove physician prescribing practices of legally marketed drugs. FDA does regulate what manufacturers may recommend about uses in their products’ labeling and what manufacturers can include in advertising and promotion.
The sometimes-used term “unapproved use” is a misnomer, implying that FDA regulates prescribing and dispensing activities. This term should be avoided.1 Other terminology that is sometimes used to describe unlabeled use includes “off-label use,” “out-of-label use,” and “usage outside of labeling.”
According to FDA, unlabeled use encompasses a range of situations that extend from inadequate to carefully conceived investigations, from hazardous to salutary uses, and from infrequent to widespread medical practice. Accepted medical practice often involves drug use that is not reflected in FDA-approved drug-product labeling.2
Health-Care Issues Related to Unlabeled Use
Access to Drug Therapies. The prescribing and dispensing of drugs for unlabeled uses are increasing.3,4 In many clinical situations, unlabeled use represents the most appropriate therapy for patients. Failure to recognize this or, more importantly, regarding such use as “unapproved” or “experimental” may restrict access to necessary drug therapies.
Lack of Practice Standards.